Assessing Treatments for Lupus Nephritis in Children

The first study comparing two therapies, mycophenolate mofetil and intravenous cyclophosphamide, for the treatment of lupus nephritis in adolescents, shows that both appear safe and effective according to research presented this week at the American College of Rheumatology Annual Scientific Meeting in San Francisco, Calif.

Lupus nephritis is an inflammation of the kidneys caused by systemic lupus erythematosus (also called SLE or lupus), which is a chronic inflammatory disease that can also affect the skin, joints, lungs, nervous system, and other organs of the body. The most commons symptoms are urinary frequency, weight gain, high blood pressure, and swelling around the eyes, legs, ankles, or fingers.

Some studies have shown that pediatric lupus is more severe and causes more kidney damage than adult-onset lupus. This recently led researchers to compare the safety and effectiveness of two immunosuppressants, which are medications that can decrease the immune response.

Specifically, researchers looked at mycophenolate mofetil (CellCept®)—a drug commonly used to help prevent organ rejection in people following kidney, liver or heart transplants—and cyclophosphamide (Cytoxan®), which belongs to a class of drugs known as alkylating agents that were originally developed and are still used to treat some types of cancer. The two drugs were compared as initial induction treatment for patients with active lupus nephritis.

Researchers followed 24 pediatric (adolescent) patients—pulled from a larger study group of 370 adult and pediatric patients who enrolled at 88 medical centers in 20 countries. Pediatric patients were predominately female, ranged in age from 12 to 17 years, and had suffered from lupus nephritis for one to three years.

Ten patients were randomly assigned to take mycophenolate mofetil by mouth; 14 patients were randomly assigned to take intravenous cyclophosphamide. Both groups received prednisone, which was tapered according to a predefined plan over the course of the study. Researchers defined successful treatment as a decrease in the concentration of protein in the urine, and stabilization or improvement in kidney function.

At week 24, nearly 88 percent of the patients remained in the study. One patient from each group withdrew, and one subject who had been treated with mycophenolate mofetil died. Of the 24 patients analyzed, 15 (62.5 percent) were considered successfully treated during the 24-week induction phase of the study. Of those, seven (70 percent successful) were taking mycophenolate mofetil and eight (57 percent successful) were taking cyclophosphamide. Serious side effects, most commonly infections, were reported in similar percentages among both groups.

In this first randomized, controlled trial involving children with lupus nephritis, researchers found that patients responded as well to mycophenolate mofetil as they did cyclophosphamide. In addition to these results, researchers noted that results in these pediatric patients were similar to those among patients enrolled in the corresponding adult study – although infections were more common among young patients than their adult counterparts.